Retrospective study of venetoclax for acute myeloid leukemia in pediatric patients

The BCL-2 inhibitor venetoclax improves survival for adult patients with acute myeloid leukemia (AML) in combination with lower-intensity therapies, but its benefit in pediatric AML patients remains unclear. A retrospective study at two centers in Houston, Texas (MD Anderson Cancer Center and Baylor College of Medicine) evaluated venetoclax-based therapy for pediatric AML. The patient population was highly refractory, with 44% having received at least 3 previous lines of therapy. The majority (86%) received venetoclax with a hypomethylating agent. The treatment was well-tolerated, with a safety profile similar to that in adults. Median EFS and OS were 3.7 months and 8.7 months, respectively. The authors conclude that more studies are needed to establish an optimal venetoclax-based regimen for the pediatric AML population.